Town Hall on Rare Diseases:Developing a Canadian Strategy for Rare Disease Treatments

(October 16, 2018) Town Hall on Rare Diseases brings together a diverse panel of speakers to discuss the challenges of providing costly drugs for Canadians suffering from rare diseases.

The public town hall, hosted by Paul Kennedy of CBC Ideas , will take place on October 23rd, 2018 , at the BMO Goldcorp Theatre in Vancouver’s Olympic Village at 6:30 p.m. Doors will open at 6:00 p.m.

This event comes after six years of work by the CIHR New Emerging Team for Rare Diseases and the Global Reporting Centre to investigate the impact of rare diseases on Canadian families, and explore options to improve access to effective therapies.

“We have consulted with a wide variety of stakeholders throughout the project, including patients and their families, clinicians, policy experts and industry representatives,” said Dr. Larry Lynd, the principal investigator for the project. “As this project nears completion, now is the time to host an informed, public discussion to continue the public dialogue on developing a strategy that will guide next steps so Canada can increase access to treatments that can save or lengthen lives.”

The Global Reporting Centre developed an award-winning multimedia documentary entitled “ Million Dollar Med$ ,” which features many of the voices that will be at the town hall, including patients, families, clinicians, patient advocates and a range of experts.

“These issues are relevant to all Canadians,” said Prof. Peter Klein, who oversaw the multimedia project and is co-organizing the town hall. “Even if you don’t have a rare disease, the money spent on these expensive drugs is coming from all of our pockets, and there are lots of questions about why these medicines are so costly, and how provinces ultimately decide who gets covered.”

One in 12 Canadians suffers from a rare disease, the majority of which are life threatening. The few diseases that do have treatments available often come with a steep price tag that the average Canadian cannot afford.

So-called “orphan drugs” comprise a growing proportion of the pharmaceutical market, with the global market worth around USD $100 billion. While provincial governments are usually willing to pay the high price for proven treatments, provincial and federal governments face an emerging dilemma of whether and how to cover these burgeoning drug costs.

The B.C. provincial government recently made such a decision to cover the cost of the newly-approveddrug Spinraza, which treats Type 1 spinal muscular atrophy, despite a multi-million dollar annual cost to treat less than 30 patients in the province.

Speakers and panelists at the event will reflect a broad diversity of voices and stakeholders in the discussion about rare diseases, treatments, and the cost, including:

● Millan Patel is a clinician-scientist, and is the research director and co-founder of the Rare Diseases Foundation. He developed a microgrant program that invests in rare disease researchprojects run by residents and fellows across Canada. Millan will provide multiple perspectives: rare disease advocate, researcher, and investor.

● Nick Harkins was diagnosed at the age of 5 with a rare metabolic disease called MPS1, which can be fatal if untreated. The only approved medication for this disease in Canada costs around $200,000 per year and only slows the progress of the disease. Nick, currently a student at UBC, will offer the perspective of a patient who has benefitted from an expensive drug.

● Dr. Sandra Sirrs is the medical director of the Adult Metabolic Diseases Clinic at Vancouver

General Hospital, one of the largest free-standing clinics for adults with metabolic disorders. She will share her perspective as a clinician who deals with the consequences of inconsistent funding/approval decisions.